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Last Updated : Jul 12, 2020 04:05 PM IST | Source: PTI

Patient support groups seek PM's intervention for finalising National Policy on Rare Diseases

LSDs and SMA are genetic and rare diseases categorised under Group 3 disorders in the draft National Policy on Rare Diseases 2020.


Patient support groups for rare diseases like Lysosomal Storage Disorders (LSDs) and Spinal Muscular Atrophy (SMA) have sought Prime Minister Narendra Modis intervention for finalising a national policy and creating an institutional funding support system towards treatment of such ailments at the earliest, president of a support group said.

LSDs and SMA are genetic and rare diseases categorised under Group 3 disorders in the draft National Policy on Rare Diseases 2020.

LSDs, in particular, impact children, causing 35 per cent of deaths below the age of 1 year, 10 per cent between 1 and 5 years and 12 per cent between 5 and 15 years, LSD Support Society (LSDSS) president Manjit Singh said.


While LSDs are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body's cells as a result of enzyme deficiencies, SMA is a genetic disorder characterized by weakness and wasting (atrophy) in muscles used for movement (skeletal muscles).

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The LSDSS and another patient support group - Cure SMA India have recently written to the Prime Minister urging him to take immediate steps for finalization of the long-pending National Policy on Rare Diseases 2020, Singh told PTI.

The Prime Minister has also been urged to take quick steps to institutionalize sustainable support mechanism towards diagnosis and treatment of Group 3 disorders in the country, at least for the ones for which the Drugs Controller General of India (DCGI) approved treatments are available, he said.

"We would like to express our gratitude to you for initiating measures to increase the government share of spending on healthcare.

"This is a step in the right direction, considering that the National Health Policy 2017 envisages raising public health expenditure progressively to 2.5 per cent of the GDP by 2025," said the representation, signed by Manjit Singh and Archana Panda, Co-Founder and Director (Patient Advocacy) of Cure SMA India.

For patients diagnosed with different types of rare diseases like LSDs and SMA, the absence of a sustained institutional support is a major hindrance, they said.

"Without any support and the inability to mobilise funds for their treatment, a majority of the patients and their care givers are left with no option but to wait for the inevitable situation and helplessly watch their children deteriorating and losing lives," they said in the representation.

They pointed out that the proposed financial support up to Rs 15 lakh as the Umbrella Scheme of Rashtriya Arogya Nidhi (RAN) to patients that require a one-time treatment (diseases listed only under Group 1) is grossly inadequate and will benefit only a small number of patients.

The patient support groups have drawn the Prime Ministers attention toward the need to have clear policy guidelines for re-appropriation of unspent funds for treatment of rare disease patients.

While over 7,000 rare diseases have been identified globally, with an extremely limited number of treatment options, in India definitive treatment is available for only 12 to 15 types of rare diseases.

Noting that the annual treatment costs for many of the patients range from Rs 50 lakh to Rs 2 crore, they said, the medical cover proposed by the draft policy will benefit only a small number of patients with rare diseases, requiring one time treatment.

"The delay in the finalization of the policy and absence of a sustainable funding mechanism for those diagnosed with rare diseases has caused immense distress to the patients.

"Majority of patients are unable to mobilise funds to support their treatment, and any further delay in the formulation of the National Policy with sustained funding mechanism will only shorten their survival prospects," Singh said.
First Published on Jul 12, 2020 03:33 pm